HHS Announces Sickle Cell Disease Initiative

Office of the Director - May 27, 2011

U.S. Department of Health and Human Services (HHS) Secretary Kathleen Sebelius announced an HHS-wide initiative to improve care for people with sickle cell disease. The announcement came during a health disparities speech Secretary Sebelius delivered in New York, drawing a round of applause from the crowd of community and public health stakeholders.

An estimated 72,000 Americans live with sickle cell disease, while approximately two million carry the sickle cell trait. Forty years ago, almost 15 percent of children born with sickle cell disease died before the age of two, and many more died in their teens. Today, the life expectancy for people with sickle cell disease has dramatically increased, and the health problems many of them experience are less severe because of new treatments that have been developed with NIH support. However, not all patients—particularly adult patients—receive state-of-the-art care, and patients of all ages need better coordinated care as well as access to health care systems and providers who have the appropriate skill sets to manage these relatively uncommon blood disorders.

With this initiative, HHS seeks to significantly improve the lives of people with sickle cell disease by building upon existing activities and increasing collaborations across government agencies. The NIH (including the NHLBI), CDC, CMS, HRSA, FDA and AHRQ will work together over the coming years to meet a set of specific needs that include:

Creating a comprehensive database of people with sickle cell disease and other blood disorders known as hemoglobinopathies. The database will help improve knowledge, clinical research recruitment, and disease management across the nation by providing information about consenting patients' ages, gender, location, access to clinical and support services, use of health care services, and health outcomes. The NHLBI and CDC have already begun a pilot project, the Registry and Surveillance of Hemoglobinopathies (RuSH), to gather population-based data from seven states.

Strengthening the evidence base for the development of sickle cell disease care guidelines and quality-of-care metrics. The NHLBI is currently developing clinical guidelines for sickle cell disease care in children and adults, including the use of hydroxyurea, to assist health care providers as well as patients and their families. The guidelines are expected to be released in spring 2012 and will be accompanied by a public awareness campaign led by the NHLBI.

Expanding high-quality "medical homes" for patients, with access to specialty care as needed.

Developing new treatments that integrate recent advances in gene therapy and targeted therapeutics.

Facilitating approval of new, safe, and effective medical products.

Enhancing the evidence base for health maintenance, health care delivery, and intervention outcomes. The NHLBI and NIH continue to support a complex portfolio of basic and translational biomedical research in sickle cell disease and thalassemia to prevent and manage complications, improve treatments, and lead to cures. Through its Clinical Science Translational Program, the NIH will also collaborate with the CDC and HRSA to research clinical implications for patients with sickle cell trait, including kidney disease, risk of blood clots, athletic participation, and access to genetic counseling.

A hematologist with a longstanding interest in sickle cell disease, NHLBI Acting Director Susan B. Shurin, M.D., has been instrumental in bringing the HHS initiative to fruition. The NHLBI looks forward to participating in this important interagency collaboration and continuing to improve the lives of people with sickle cell disease and related conditions.